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Article: Current understanding of dystrophin-related muscular dystrophy and therapeutic challenges ahead

TitleCurrent understanding of dystrophin-related muscular dystrophy and therapeutic challenges ahead
Authors
Issue Date2006
PublisherChinese Medical Association. The Journal's web site is located at http://www.cmj.org/
Citation
Chinese Medical Journal, 2006, v. 119 n. 16, p. 1381-1391 How to Cite?
AbstractObjective: To review the recent, research progress in dystrophin-related muscular dystrophy includes X-linked hereditary Duchenne and Becker muscular dystrophies (DMD and BMD). Data sources: Information included in this article was identified by searches of PUBMED and other online resources using the key terms DMD, dystrophin, mutations, animal models, pathophysiology, gene expression, stem cells, gene therapy, cell therapy, and pharmacological. Study selection: Mainly original milestone articles and timely reviews written by major pioneer investigators of the field were selected. Results: The key issues related to the genetic basis and pathophysiological factors of the diseases were critically addressed. The availabilities and advantages of various animal models for the diseases were described. Major molecular and cellular therapeutic approaches were also discussed, many of which have indeed exhibited some success in pre-clinical studies but at the same time encountered a number of technical hurdles, including the efficient and systemic delivery of a functional gene and myogenic precursor/stem cells to repair genetic defects. Conclusions: Further understanding of pathophysiological mechanisms at molecular levels and regenerative properites of myogenic precursor/stem cells will promote the development of multiple therapeutic strategies. The combined use of multiple strategies may represent the major challenge as well as the greatest hope for the therapy of these diseases in coming years.
Persistent Identifierhttp://hdl.handle.net/10722/170090
ISSN
2015 Impact Factor: 0.957
2015 SCImago Journal Rankings: 0.428
ISI Accession Number ID
References

 

DC FieldValueLanguage
dc.contributor.authorZhou, GQen_US
dc.contributor.authorXie, HQen_US
dc.contributor.authorZhang, SZen_US
dc.contributor.authorYang, ZMen_US
dc.date.accessioned2012-10-30T06:05:15Z-
dc.date.available2012-10-30T06:05:15Z-
dc.date.issued2006en_US
dc.identifier.citationChinese Medical Journal, 2006, v. 119 n. 16, p. 1381-1391en_US
dc.identifier.issn0366-6999en_US
dc.identifier.urihttp://hdl.handle.net/10722/170090-
dc.description.abstractObjective: To review the recent, research progress in dystrophin-related muscular dystrophy includes X-linked hereditary Duchenne and Becker muscular dystrophies (DMD and BMD). Data sources: Information included in this article was identified by searches of PUBMED and other online resources using the key terms DMD, dystrophin, mutations, animal models, pathophysiology, gene expression, stem cells, gene therapy, cell therapy, and pharmacological. Study selection: Mainly original milestone articles and timely reviews written by major pioneer investigators of the field were selected. Results: The key issues related to the genetic basis and pathophysiological factors of the diseases were critically addressed. The availabilities and advantages of various animal models for the diseases were described. Major molecular and cellular therapeutic approaches were also discussed, many of which have indeed exhibited some success in pre-clinical studies but at the same time encountered a number of technical hurdles, including the efficient and systemic delivery of a functional gene and myogenic precursor/stem cells to repair genetic defects. Conclusions: Further understanding of pathophysiological mechanisms at molecular levels and regenerative properites of myogenic precursor/stem cells will promote the development of multiple therapeutic strategies. The combined use of multiple strategies may represent the major challenge as well as the greatest hope for the therapy of these diseases in coming years.en_US
dc.languageengen_US
dc.publisherChinese Medical Association. The Journal's web site is located at http://www.cmj.org/en_US
dc.relation.ispartofChinese Medical Journalen_US
dc.subject.meshAnimalsen_US
dc.subject.meshDisease Models, Animalen_US
dc.subject.meshDystrophin - Genetics - Physiologyen_US
dc.subject.meshGene Therapy - Methodsen_US
dc.subject.meshHumansen_US
dc.subject.meshModels, Biologicalen_US
dc.subject.meshMuscular Dystrophies - Genetics - Physiopathology - Therapyen_US
dc.subject.meshMutation - Geneticsen_US
dc.subject.meshUtrophin - Therapeutic Useen_US
dc.titleCurrent understanding of dystrophin-related muscular dystrophy and therapeutic challenges aheaden_US
dc.typeArticleen_US
dc.identifier.emailZhou, GQ:wormoscz@gmail.comen_US
dc.identifier.authorityZhou, GQ=rp00527en_US
dc.description.naturelink_to_OA_fulltexten_US
dc.identifier.pmid16934185-
dc.identifier.scopuseid_2-s2.0-33747637780en_US
dc.relation.referenceshttp://www.scopus.com/mlt/select.url?eid=2-s2.0-33747637780&selection=ref&src=s&origin=recordpageen_US
dc.identifier.volume119en_US
dc.identifier.issue16en_US
dc.identifier.spage1381en_US
dc.identifier.epage1391en_US
dc.identifier.isiWOS:000240154100011-
dc.publisher.placeChinaen_US
dc.identifier.scopusauthoridZhou, GQ=23394245100en_US
dc.identifier.scopusauthoridXie, HQ=7401672194en_US
dc.identifier.scopusauthoridZhang, SZ=23053513900en_US
dc.identifier.scopusauthoridYang, ZM=7405433260en_US

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